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Autosomal recessive titinopathy

MedGen UID:
993943
Concept ID:
CN315649
Disease or Syndrome
Synonyms: autosomal recessive titinopathy; TTN-related myopathy, autosomal recessive
 
Monarch Initiative: MONDO:0100493

Definition

Autosomal recessive form of TTN-related myopathy. [from MONDO]

Recent clinical studies

Etiology

Meyer AP, Barnett CL, Myers K, Siskind CE, Moscarello T, Logan R, Roggenbuck J, Rich KA
J Med Genet 2024 Mar 21;61(4):356-362. doi: 10.1136/jmg-2023-109513. PMID: 38050027
Khan A, Wang R, Han S, Umair M, Abbas S, Khan MI, Alshabeeb MA, Alfadhel M, Zhang X
BMC Med Genet 2019 Oct 29;20(1):166. doi: 10.1186/s12881-019-0895-7. PMID: 31664938Free PMC Article
Guglieri M, Straub V, Bushby K, Lochmüller H
Curr Opin Neurol 2008 Oct;21(5):576-84. doi: 10.1097/WCO.0b013e32830efdc2. PMID: 18769252

Diagnosis

Meyer AP, Barnett CL, Myers K, Siskind CE, Moscarello T, Logan R, Roggenbuck J, Rich KA
J Med Genet 2024 Mar 21;61(4):356-362. doi: 10.1136/jmg-2023-109513. PMID: 38050027
Perrin A, Métay C, Savarese M, Ben Yaou R, Demidov G, Nelson I, Solé G, Péréon Y, Bertini ES, Fattori F, D'Amico A, Ricci F, Ginsberg M, Seferian A, Boespflug-Tanguy O, Servais L, Chapon F, Lagrange E, Gaudon K, Bloch A, Ghanem R, Guyant-Maréchal L, Johari M, Van Goethem C, Fardeau M, Morales RJ, Genetti CA, Marttila M, Koenig M, Beggs AH, Udd B, Bonne G, Cossée M
J Med Genet 2024 Mar 21;61(4):369-377. doi: 10.1136/jmg-2023-109473. PMID: 37935568Free PMC Article
Bryen SJ, Ewans LJ, Pinner J, MacLennan SC, Donkervoort S, Castro D, Töpf A, O'Grady G, Cummings B, Chao KR, Weisburd B, Francioli L, Faiz F, Bournazos AM, Hu Y, Grosmann C, Malicki DM, Doyle H, Witting N, Vissing J, Claeys KG, Urankar K, Beleza-Meireles A, Baptista J, Ellard S, Savarese M, Johari M, Vihola A, Udd B, Majumdar A, Straub V, Bönnemann CG, MacArthur DG, Davis MR, Cooper ST
Hum Mutat 2020 Feb;41(2):403-411. Epub 2019 Dec 3 doi: 10.1002/humu.23938. PMID: 31660661Free PMC Article
Perić S, Glumac JN, Töpf A, Savić-Pavićević D, Phillips L, Johnson K, Cassop-Thompson M, Xu L, Bertoli M, Lek M, MacArthur D, Brkušanin M, Milenković S, Rašić VM, Banko B, Maksimović R, Lochmüller H, Stojanović VR, Straub V
Eur J Hum Genet 2017 May;25(5):572-581. Epub 2017 Mar 15 doi: 10.1038/ejhg.2017.16. PMID: 28295036Free PMC Article
Guglieri M, Straub V, Bushby K, Lochmüller H
Curr Opin Neurol 2008 Oct;21(5):576-84. doi: 10.1097/WCO.0b013e32830efdc2. PMID: 18769252

Prognosis

Meyer AP, Barnett CL, Myers K, Siskind CE, Moscarello T, Logan R, Roggenbuck J, Rich KA
J Med Genet 2024 Mar 21;61(4):356-362. doi: 10.1136/jmg-2023-109513. PMID: 38050027
McDermott H, Henderson A, Robinson HK, Heaver R, Halahakoon C, Cox H, Naik S
Neuromuscul Disord 2021 Aug;31(8):783-787. Epub 2021 May 24 doi: 10.1016/j.nmd.2021.05.004. PMID: 34303570
Chervinsky E, Khayat M, Soltsman S, Habiballa H, Elpeleg O, Shalev S
Am J Med Genet A 2018 Apr;176(4):1001-1005. doi: 10.1002/ajmg.a.38639. PMID: 29575618
Guglieri M, Straub V, Bushby K, Lochmüller H
Curr Opin Neurol 2008 Oct;21(5):576-84. doi: 10.1097/WCO.0b013e32830efdc2. PMID: 18769252

Clinical prediction guides

Meyer AP, Barnett CL, Myers K, Siskind CE, Moscarello T, Logan R, Roggenbuck J, Rich KA
J Med Genet 2024 Mar 21;61(4):356-362. doi: 10.1136/jmg-2023-109513. PMID: 38050027

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